CIRM-funded treatment for cancer granted FDA breakthrough therapy designation

Mark Chao, M.D., Ph.D., cofounder of Forty Seven, Inc. and current VP of oncology clinical research at Gilead Sciences

An antibody therapeutic, magrolimab, being tested for myelodysplastic syndrome (MDS), a group of cancers in which the bone marrow does not produce enough healthy blood cells , was granted breakthrough therapy designation with the Food and Drug Administration (FDA). 

Breakthrough therapy designations from the FDA are intended to help expedite the development of new treatments. They require preliminary clinical evidence that demonstrates that the treatment may have substantial improvement in comparison to therapy options currently available. CIRM funded a Phase 1b trial in MDS and acute myeloid leukemia (AML), another type of blood cancer, that provided the data on which the breakthrough therapy designation is based.

Cancer cells express a signal known as CD47, which sends a “don’t eat me” message to macrophages, white blood cells that are part of the immune system designed to “eat” and destroy unhealthy cells. Magrolimab works by blocking the signal, enabling the body’s own immune system to detect and destroy the cancer cells.

Magrolimab was initially developed by a team led by Irv Weissman, M.D. at Stanford University with the support of CIRM awards. This led to the formation of Forty Seven, Inc., which was subsequently acquired by Gilead Sciences in April 2020 for $4.9 billion (learn more about other highlighted partnership events on CIRM’s Industry Alliance Program website by clicking here).

In CIRM’s 2019-2020 18-Month Report, Mark Chao, M.D., Ph.D.,  who co-founded Forty Seven, Inc. and currently serves as the VP of oncology clinical research at Gilead Sciences, credits CIRM with helping progress this treatment.

“CIRM’s support has been instrumental to our ability to rapidly progress Forty Seven’s CD47 antibody targeting approach.”

Magrolimab is currently being studied as a combination therapy with azacitidine, a chemotherapy drug, in a Phase 3 clinical trial in previously untreated higher risk MDS. This is one of the last steps before seeking FDA approval for widespread commercial use.

Merdad Parsey, MD, PhD, Chief Medical Officer at Gilead Sciences

In a press release, Merdad Parsey, M.D., Ph.D., Chief Medical Officer at Gilead Sciences discusses the significance of the designation from the FDA and the importance of the treatment.

“The Breakthrough Therapy designation recognizes the potential for magrolimab to help address a significant unmet medical need for people with MDS and underscores the transformative potential of Gilead’s immuno-oncology therapies in development.”

CIRM-funded life-saving stem cell therapy gets nod of approval from FDA

Cured_AR_2016_coverIf you have read our 2016 Annual Report (and if you haven’t you should, it’s brilliant) or just seen the cover you’ll know that it features very prominently a young girl named Evie Padilla Vaccaro.

Evie was born with Severe Combined Immunodeficiency or SCID – also known as “bubble baby disease”; we’ve written about it here. SCID is a rare but deadly immune disorder which leaves children unable to fight off simple infections. Many children with SCID die in the first few years of life.

Fortunately for Evie and her family, Dr. Don Kohn and his team at UCLA, working with a UK-based company called Orchard Therapeutics Ltd., have developed a treatment called OTL-101. This involves taking the patient’s own blood stem cells, genetically modifying them to correct the SCID mutation, and then returning the cells to the patient. Those modified cells create a new blood supply, and repair the child’s immune system.

Evie was treated with OTL-101 when she was a few months old. She is cured. And she isn’t the only one. To date more than 40 children have been treated with this method. All have survived and are doing well.

Orchard Therapeutics

 FDA acknowledgement

Because of that success the US Food and Drug Administration (FDA) has granted OTL-101 Rare Pediatric Disease Designation. This status is given to a treatment that targets a serious or life-threatening disease that affects less than 200,000 people, most of whom are under 18 years of age.

The importance of the Rare Pediatric Disease Designation is that it gives the company certain incentives for the therapy’s development, including priority review by the FDA. That means if it continues to show it is safe and effective it may have a faster route to being made more widely available to children in need.

In a news release Anne Dupraz, PhD, Orchard’s Chief Regulatory Officer, welcomed the decision:

“Together with Orphan Drug and Breakthrough Therapy Designations, this additional designation is another important development step for the OTL-101 clinical program. It reflects the potential of this gene therapy treatment to address the significant unmet medical need of children with ADA-SCID and eligibility for a Pediatric Disease Priority Review voucher at time of approval.”

Creating a trend

This is the second time in less than two weeks that a CIRM-funded therapy has been awarded Rare Pediatric Disease designation. Earlier this month Capricor Therapeutics was given that status for its treatment for Duchenne Muscular Dystrophy.

Two other CIRM-funded clinical trials – Humacyte and jCyte – have been given Regenerative Medicine Advanced Therapy Designation (RMAT) by the FDA. This makes them eligible for earlier and faster interactions with the FDA, and also means they may be able to apply for priority review and faster approval.

All these are encouraging signs for a couple of reasons. It suggests that the therapies are showing real promise in clinical trials. And it shows that the FDA is taking steps to encourage those therapies to advance as quickly – and safely of course – as possible.

Credit where credit is due

In the past we have been actively critical of the FDA’s sluggish pace in moving stem cell therapies out of the lab and into clinical trials where they can be tested in people. So when the FDA does show signs of changing the way it works it’s appropriate that that we are actively supportive.

Getting these designations is, of course, no guarantee the therapies will ultimately prove to be successful. But if they are, creating faster pathways means they can get to patients, the people who really need them, at a much faster pace.